{"id":113028,"date":"2020-09-15T13:24:29","date_gmt":"2020-09-15T20:24:29","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2020\/09\/twist-on-crispr-gene-editing-treats-adult-onset-muscular-dystrophy-in-mice"},"modified":"2020-09-15T13:24:29","modified_gmt":"2020-09-15T20:24:29","slug":"twist-on-crispr-gene-editing-treats-adult-onset-muscular-dystrophy-in-mice","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2020\/09\/twist-on-crispr-gene-editing-treats-adult-onset-muscular-dystrophy-in-mice","title":{"rendered":"Twist on CRISPR Gene Editing Treats Adult-Onset Muscular Dystrophy in Mice"},"content":{"rendered":"<p><a class=\"aligncenter blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/twist-on-crispr-gene-editing-treats-adult-onset-muscular-dystrophy-in-mice2.jpg\"><\/a><\/p>\n<p>Myotonic dystrophy type I is the most common type of adult-onset muscular dystrophy. People with the condition inherit repeated DNA segments that lead to the toxic buildup of repetitive RNA, the messenger that carries a gene\u2019s recipe to the cell\u2019s protein-making machinery. As a result, people born with myotonic dystrophy experience progressive muscle wasting and weakness and a wide variety of other debilitating symptoms.<\/p>\n<p>CRISPR-Cas9 is a technique increasingly used in efforts to correct the genetic (DNA) defects that cause a variety of diseases. A few years ago, University of California San Diego School of Medicine researchers redirected the technique to instead modify RNA in a method they call RNA-targeting Cas9 (RCas9).<\/p>\n<p>In a new study, publishing September 14, 2020 in <a href=\"https:\/\/www.nature.com\/articles\/s41551-020-00607-7\" target=\"_blank\"><em>Nature Biomedical Engineering<\/em><\/a>, the team demonstrates that one dose of RCas9 gene therapy can chew up toxic RNA and almost completely reverse symptoms in a mouse model of myotonic dystrophy.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Myotonic dystrophy type I is the most common type of adult-onset muscular dystrophy. People with the condition inherit repeated DNA segments that lead to the toxic buildup of repetitive RNA, the messenger that carries a gene\u2019s recipe to the cell\u2019s protein-making machinery. As a result, people born with myotonic dystrophy experience progressive muscle wasting and [\u2026]<\/p>\n","protected":false},"author":591,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1902,11,412],"tags":[],"class_list":["post-113028","post","type-post","status-publish","format-standard","hentry","category-bioengineering","category-biotech-medical","category-genetics"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/113028","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/591"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=113028"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/113028\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=113028"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=113028"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=113028"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}